Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation

Usher syndrome (USH) is the most common cause of combined deaf-blindness in man. The hearing loss can be partly compensated by providing patients with hearing aids or cochlear implants, but the loss of vision is currently untreatable. In general, mutations in the USH2A gene are the most frequent cau...

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Главные авторы:	Radulfus WN Slijkerman, Christel Vaché, Margo Dona, Gema García-García, Mireille Claustres, Lisette Hetterschijt, Theo A Peters, Bas P Hartel, Ronald JE Pennings, José M Millan, Elena Aller, Alejandro Garanto, Rob WJ Collin, Hannie Kremer, Anne-Françoise Roux, Erwin Van Wijk
Формат:	Artigo
Язык:	Inglês
Опубликовано:	Elsevier 2016-01-01
Серии:	Molecular Therapy: Nucleic Acids
Предметы:	antisense oligonucleotides genetic therapy splice redirection USH2A Usher syndrome
Online-ссылка:	http://www.sciencedirect.com/science/article/pii/S2162253117301087
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